On the normativity of evidence - Lessons from philosophy of science and the "VALIDATE" project.

"Evidence" is a key term in medicine and health services research, including Health Technology Assessment (HTA). Randomized clinical trials (RCTs) have undoubtedly dominated the scene of generating evidence for a long period of time, becoming the hallmark of evidence-based medicine (EBM). However, due to a number of misunderstandings, the lay audience and some researchers have sometimes placed too much trust in RCTs compared to other methods of investigation. One of the principal misunderstandings is to consider RCTs findings as isolated and self-apparent pieces of information. In other words, what has been essentially lacking was the awareness of the value-context of the evidence and, in particular, the value- and theory-ladenness (normativity) of scientific knowledge. This paper aims to emphasize the normativity that exists in the production of scientific knowledge, and in particular in the conduct of RCTs as well as in the performance of HTA. The work is based on some lessons learned from Philosophy of Science and the European project "VALIDATE" (VALues In Doing Assessments of healthcare TEchnologies"). VALIDATE was a three-year EU Erasmus+ strategic partnerships project (2018-2021), in which training in the field of HTA was further optimized by using insights from political science and ethics (in accordance with the recent definition of HTA). Our analysis may reveal useful insights for addressing some challenges that HTA is going to face in the future.

Implementing a Rapid Response System in a tertiary-care hospital. A cost-effectiveness study.

PURPOSE: The occurrence of adverse events (AE) in hospitalized patients substancially increases the risk of disability or death, having a major negative clinical and economic impact on public health. For early identification of patients at risk and to establish preventive measures, different healthcare systems have implemented rapid response systems (RRS). The aim of this study was to carry out a cost-effectiveness analysis of implementing a RRS in a tertiary-care hospital. METHODS: We included all the patients admitted to Hospital Clínic de Barcelona from 1 to 2016 to 31 December 2016. The cost-effectiveness analysis was summarized as the incremental cost-effectiveness ratio (incremental cost divided by the incremental effectiveness of the two alternatives, RRS versus non-RRS). The effectiveness of the RRS, defined as improvements in health outcomes (AE, cardiopulmonary arrest and mortality), was obtained from the literature and applied to the included patient cohort. A budget impact analysis on the implementation of the RRS from a hospital perspective was performed over a 5-year time horizon. RESULTS: 42,409 patients were included, and 448 (1.05%) had severe AE requiring ICU admission. The cost-effectiveness analysis showed an incremental cost (savings) of EUR - 1,471,101 of RRS versus the non-RRS. The budgetary impact showed a cost reduction of EUR 896,762.00 in the first year and EUR 1,588,579.00 from the second to the fifth year. CONCLUSIONS: The present analysis shows the RRS as a dominant, less costly and more effective structure compared to the non-RRS.

New regulation of medical devices in the EU: impact in dermatology.

Medical device (MD) is a broad term that encompasses products ranging from, for example, examination gloves to digital dermoscopy systems; all of which are regulated by a new regulatory framework in the EU from May 2021. The new Medical Device Regulation (MDR) (Regulation EU 2017/745) will have a significant effect on suppliers of MD and will have subsequent effects also for dermatologists and other clinicians. Medical device software and apps are reclassified leading to more stringent requirements on documentation within, e.g. clinical evidence, as well as regulatory authority control. The changes will likely have positive effects on quality, to the benefit of patients. There will, however, be implications affecting the availability and support of existing devices and the introduction of new devices, as well as a likely price increase due to the higher costs for suppliers. Dermatologists, other clinicians and administrators need to be aware of the effects of MDR to ensure that existing devices and new purchases can be used as planned. Specifically, clinicians need to be aware of the following: (i) improved quality of MD and follow-up of incidents can be expected. (ii) Only 'non-significant' updates will be permitted after May 2021 to many existing systems and devices unless approved under the new MDR. (iii) Existing devices that do not achieve approval under the new regulation will no longer be manufactured after May 2024. (iv) New products and methods will take longer time to be approved and available. (v) Prices will likely increase. (vi) Suppliers of products that do not fulfil the new regulation will disappear, and the availability of consumables, spare parts or upgrades might be discontinued. (vii) A trend to oligopoly may appear in the market. It is therefore important to check with your suppliers as to how and when they will adhere to the new MDR regulation.

PrediCTC, liquid biopsy in precision oncology: a technology transfer experience in the Spanish health system

Purpose. Management of metastatic disease in oncology includes monitoring of therapy response principally by imaging techniques like CT scan. In addition to some limitations, the irruption of liquid biopsy and its application in personalized medicine has encouraged the development of more efficient technologies for prognosis and follow-up of patients in advanced disease. Methods. PrediCTC constitutes a panel of genes for the assessment of circulating tumor cells (CTC) in metastatic colorectal cancer patients, with demonstrated improved efficiency compared to CT scan for the evaluation of early therapy response in a multicenter prospective study. In this work, we designed and developed a technology transfer strategy to define the market opportunity for an eventual implementation of PrediCTC in the clinical practice. Results. This included the definition of the regulatory framework, the analysis of the regulatory roadmap needed for CE mark, a benchmarking study, the design of a product development strategy, a revision of intellectual property, a cost-effectiveness study and an expert panel consultation. Conclusion. The definition and analysis of an appropriate technology transfer strategy and the correct balance among regulatory, financial and technical determinants are critical for the transformation of a promising technology into a viable technology, and for the decision of implementing liquid biopsy in the monitoring of therapy response in advanced disease

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PrediCTC, liquid biopsy in precision oncology: a technology transfer experience in the Spanish health system.

PURPOSE: Management of metastatic disease in oncology includes monitoring of therapy response principally by imaging techniques like CT scan. In addition to some limitations, the irruption of liquid biopsy and its application in personalized medicine has encouraged the development of more efficient technologies for prognosis and follow-up of patients in advanced disease. METHODS: PrediCTC constitutes a panel of genes for the assessment of circulating tumor cells (CTC) in metastatic colorectal cancer patients, with demonstrated improved efficiency compared to CT scan for the evaluation of early therapy response in a multicenter prospective study. In this work, we designed and developed a technology transfer strategy to define the market opportunity for an eventual implementation of PrediCTC in the clinical practice. RESULTS: This included the definition of the regulatory framework, the analysis of the regulatory roadmap needed for CE mark, a benchmarking study, the design of a product development strategy, a revision of intellectual property, a cost-effectiveness study and an expert panel consultation. CONCLUSION: The definition and analysis of an appropriate technology transfer strategy and the correct balance among regulatory, financial and technical determinants are critical for the transformation of a promising technology into a viable technology, and for the decision of implementing liquid biopsy in the monitoring of therapy response in advanced disease.

Estudio coste-beneficio del trasplante de membrana amniótica para úlceras venosas de extremidades inferiores refractarias a tratamiento convencional / Cost-Benefit Analysis of Amniotic Membrane Transplantation for Venous Ulcers of the Legs That Are Refractory to Conventional Treatment

Introducción: El tratamiento estándar compresivo de las úlceras venosas de extremidades inferiores no promueve la cicatrizacion. El autoinjerto es uno de los tratamientos que acelera la reparación tisular, sin embargo, su aplicación es difícil en pacientes pluripatológicos o cuando se requieren aplicaciones múltiples. La membrana amniótica se ha empleado como material de cobertura y como epitelizante, por lo que podría ser una buena opción terapéutica para cuando el autoinjerto no esté indicado. Objetivos: Analizar el estado de conocimiento científico sobre la seguridad y la eficacia de la membrana amniótica y comparar los costes de los injertos disponibles actualmente (autoinjertos, membrana amniótica y sustitutos cutáneos biocompatibles) para promover la reparación tisular de las úlceras venosas. Material y métodos: Se realizó una búsqueda y revisión sistemática de la literatura científica hasta marzo del 2010 sobre el uso de la membrana amniótica como tratamiento de las úlceras venosas. Asimismo, se realizó un análisis coste-minimización (horizonte temporal 3 y 6 meses). Se consideraron los costes directos sanitarios. Para comprobar la estabilidad de los resultados se llevó a cabo un análisis de sensibilidad. Resultados: Se identificó un único estudio sobre seguridad y eficacia. El análisis de costes mostró que el autoinjerto es siempre la opción más barata (1.053 D versus 1.825 D membrana amniótica, 5.767 D sustitutos cutáneos biocompatibles). A los 6 meses la membrana amniótica costaría 6.765 D menos que el uso de los sustitutos cutáneos biocompatibles. Conclusiones: El transplante de membrana amniótica para la reepitelización de úlceras venosas refractarias al tratamiento convencional es una opción terapéutica de gran potencial, pero aúnen estado experimental. El autoinjerto es el tratamiento más eficiente; pero la membrana amniótica es más económica que los sustitutos cutáneos biocompatibles (AU)

Background: Standard compression therapy for venous ulcers of the legs does not promote healing. Although autografting accelerates tissue repair, it is difficult to use in patients with concomitant diseases or when multiple grafts are required. The amniotic membrane has been used as a covering material and promotes epithelialization, making it a good potential treatment option when autografts are not indicated. Objectives: To analyze the literature on the safety and efficacy of amniotic membrane grafting and compare the cost of currently available grafts (autografts, amniotic membrane grafts, and biocompatible skin substitutes) to promote tissue repair in venous ulcers. Material and methods: A systematic review of the literature on the use of amniotic membrane grafts for the treatment of venous ulcers was performed up to 2010. A cost-minimization analysis of direct healthcare costs was then performed (at 3 and 6 months). A sensitivity analysis was performed to confirm the stability of the results. Results: Only 1 study addressing safety and efficacy was identified. The cost-minimization analysis showed that autografts are always the least-expensive option (D 1053 compared with D 1825 for amniotic membrane grafts and D 5767 for biocompatible skin grafts). At 6 months, however, amniotic membrane grafts would have cost D 6765 less than the use of biocompatible skin substitutes. Conclusions: Despite having excellent therapeutic potential for the re-epithelialization of venous ulcers that do not respond to conventional treatment, amniotic membrane transplant remains an experimental therapy. Autograft is the most efficient treatment but amniotic membrane graft is less expensive than the use of biocompatible skin substitutes (AU)

[Cost-benefit analysis of amniotic membrane transplantation for venous ulcers of the legs that are refractory to conventional treatment]. / Estudio coste-beneficio del trasplante de membrana amniótica para úlceras venosas de extremidades inferiores refractarias a tratamiento convencional.

BACKGROUND: Standard compression therapy for venous ulcers of the legs does not promote healing. Although autografting accelerates tissue repair, it is difficult to use in patients with concomitant diseases or when multiple grafts are required. The amniotic membrane has been used as a covering material and promotes epithelialization, making it a good potential treatment option when autografts are not indicated. OBJECTIVES: To analyze the literature on the safety and efficacy of amniotic membrane grafting and compare the cost of currently available grafts (autografts, amniotic membrane grafts, and biocompatible skin substitutes) to promote tissue repair in venous ulcers. MATERIAL AND METHODS: A systematic review of the literature on the use of amniotic membrane grafts for the treatment of venous ulcers was performed up to 2010. A cost-minimization analysis of direct healthcare costs was then performed (at 3 and 6 months). A sensitivity analysis was performed to confirm the stability of the results. RESULTS: Only 1 study addressing safety and efficacy was identified. The cost-minimization analysis showed that autografts are always the least-expensive option (€ 1053 compared with € 1825 for amniotic membrane grafts and € 5767 for biocompatible skin grafts). At 6 months, however, amniotic membrane grafts would have cost € 6765 less than the use of biocompatible skin substitutes. CONCLUSIONS: Despite having excellent therapeutic potential for the re-epithelialization of venous ulcers that do not respond to conventional treatment, amniotic membrane transplant remains an experimental therapy. Autograft is the most efficient treatment but amniotic membrane graft is less expensive than the use of biocompatible skin substitutes.

Wide social participation in prioritizing patients on waiting lists for joint replacement: a conjoint analysis.

OBJECTIVE: The aim was to develop a priority scoring system for patients on waiting lists for joint replacement based on a wide social participation, and to analyze the differences among participants. METHODS: Conjoint analysis. Focus groups in combination with a nominal technique were employed to identify the priority criteria (N=36). A rank-ordered logit model was then applied for scoring estimations. Participants (N=860) represented: consultants, allied-health professionals, patients and their relatives, and the general population of Catalonia. RESULTS: Clinical and social criteria were selected, and their relative importance (over 100 points) was: pain (33), difficulty in doing activities of daily living (21), disease severity (18), limitations on ability to work (10), having someone to look after the patient (9), being a caregiver (6), and recovery probability (4). Estimated criteria coefficients had the expected positive sign and all were statistically significant (P < 0.001). There were differences between groups; pain was rated higher by patients/relatives, and difficulty in doing activities was rated lower by patients/relatives and the general public. Most interaction terms for these criteria and groups were significant (P < 0.001). Consultants and allied-health professionals had the most similar prioritization pattern (r=0.97). CONCLUSION: Both clinical and social criteria are considered for prioritization of joint replacement surgery from a wide social perspective. The preference among professional and social groups varies and this might impact the result of patient prioritization. A wide social participation for obtaining adequate prioritizing systems for patients on waiting lists is desirable.

[Citizens opinions, experiences and perceptions about waiting lists for elective cataract surgery and hip and knee replacement. ]. / Opiniones, vivencias y percepciones de los ciudadanos en torno a las listas de espera para cirugía electiva de catarata y artroplastia de cadera y rodilla.

OBJECTIVES: The aim of this study is to describe the views, perceptions and personal experience around elective cataract and hip and knee replacement waiting lists in Catalonia in order to improve their management. DESIGN: Qualitative methodology: four focus groups for each procedure. PLACE: Conducted between February-March 2000 at the Catalan Health Services headquarters. PARTICIPANTS: Each focus group included consultants (ophtalmologists, orthopedic surgeons, rheumatologists, rehabilitators, GPs), other related health professionals (nurses, social workers, physiotherapists, opticians/optometrist, GPs), patients and relatives and general population. Participants were selected through researchers and the clinical scientific committees. All of them followed a pre-established inclusion criteria. MAIN MEASURES: The analysis of the information was performed using the content analysis technique (contents of sessions were transcripted and information classified according to themes). RESULTS: Ten themes were identified. Waiting lists were argued to be a consequence of lack of resources, bad management and conflict of interest among consultants. Overall, the health care authority responsibility for the solution was acknowledged, although some participants claim more citizen participation. Among proposed solutions, prevention and education, more resources and improved management were found. Furthermore, a better physician-patient communication was considered essential. All the groups disagreed with the implicit current prioritisation system, however none wanted to assume the responsibility. CONCLUSIONS: Citizen's perceptions and personal experiences point to a multifactorial approach to waiting lists management, which would ameliorate the problem and lead to a better social acceptance.

Evaluación de tecnología médica en el ámbito hospitalario. Aplicación al diagnóstico por la imagen / No disponible

Los avances tecnológicos en el área del diagnóstico por la imagen llevados a cabo en las últimas décadas han contribuido de forma significativa al diagnóstico, y manejo de un gran número de patologías. Las técnicas diagnósticas no sólo proporcionan hoy soporte al diagnóstico clínico, sino que también se aproximan más al paciente mediante la aplicación de técnicas diagnósticas invasivas (p. ej. biopsia guiada) y terapéuticas (p. ej. tratamiento con radiofármacos). No obstante, estos avances junto con el rápido crecimiento de la industria sanitaria y la globalización y fácil acceso a todo tipo de información, están contribuyendo a generar una demanda creciente clínica y social por la adquisición de la última y más novedosa tecnología médica (TM). Debe explicitarse que bajo el término de TM se incluyen equipos, fármacos, prótesis, procedimientos médi¬cos y quirúrgicos, susceptibles de ser utilizados por los profesionales de la medicina para la prevención, diagnósti¬co, tratamiento y rehabilitación de condiciones clínicas con¬cretas; también se incluyen los sistemas de información y apoyo a la atención sanitaria (AU)

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Identifying bone-mass-related risk factors for fracture to guide bone densitometry measurements: a systematic review of the literature.

Available evidence suggests that fracture prediction with bone densitometry may improve when used on people at high risk of osteoporotic fractures. The objectives of this literature review were: (1) to identify risk factors for fracture that are associated with the development of a low bone mass for both men and women; (2) to describe and assess the relationship between these factors and the risk of fracture; and (3) to classify them according to the strength of their association with fracture incidence. Studies were identified from MEDLINE (1982-1997), HealthSTAR (1975-1997) and The Cochrane Library (1997) databases. Pre-stated inclusion criteria (original analytic studies assessing risk factors for osteoporotic fractures in men and women) and methodologic quality were assessed by two independent investigators. Information on the study design and analysis, characteristics of participants, exposure (risk factor) and outcome measures (relative risk and odds ratios for fracture incidence), control for potential confounding factors and risk estimates was extracted using a standardized protocol. Qualitative and meta-analytic techniques were used for data synthesis. As a result, risk factors were classified into three groups according to their strength of association with fracture: high risk (RR > or = 2), moderate risk (1 < RR < 2) and no risk or protective (RR < or = 1). Of approximately 80 risk factors identified from 94 cohort and 72 case-control studies, 15% were classified in the high-risk group, including low body weight, loss of weight, physical inactivity, the consumption of corticosteroids or anticonvulsants, primary hyperparathyroidism, diabetes mellitus type 1, anorexia nervosa, gastrectomy, pernicious anemia, and aging (> 70-80 years). Eighteen percent and 8% of risk factors were classified in the moderate and no risk group respectively, whereas 60% showed either a lack of scientific evidence confirming their association with fracture or contradictory results. An efficient strategy for bone densitometry provision may thus be its selective use in those individuals who present with several strong or moderate risk factors for fracture related to bone mass loss.

Health technology assessment in Spain.

The Spanish Constitution of 1978 established a healthcare system available to everyone and free at the point of service. The General Health Law of 1986 also established the framework for a National Health System (NHS). The Constitution and the law form the regulatory framework for the devolution of healthcare services to the Autonomous Regions. All the 17 Autonomous Regions have complete power regarding public health and planning. However, responsibilities on healthcare financing, organization, provision, and management have devolved to only seven Autonomous Regions. Financial support for health services comes mostly from taxes. Global budgets are a mechanism used by hospitals to control the acquisition of medium and low health technology. Major capital investments for health technology are controlled by the central government in 10 Autonomous Regions (population coverage of 38%) and by the Regional Health Services in the seven remaining Autonomous Regions. In 1995 a regulation for basing the introduction of new procedures and medical equipment on the assessment of safety, efficacy, and efficiency was issued. Health technology assessment (HTA) has a long history in Spain, beginning with the Advisory Board on High Technology in the government of Catalonia in 1984. This board evolved into the Catalan Agency for HTA (CAHTA) in 1994. The Basque Country established a unit for HTA in 1992 (Osteba) and the Andalusian government created an agency in 1996 (AETSA). A national agency for HTA (AETS) was established in 1994. These different programs coordinate their work and together act as an Advisory Committee of the Interregional Council of the NHS.

Evaluación de tecnología médica en el ámbito hospitalario. Aplicación al diagnóstico por la imagen / Evaluation of Medical Technology in the Hospital Field as Applied to Diagnosis by images

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The effectiveness of bone density measurement and associated treatments for prevention of fractures. An international collaborative review.

This review assesses the evidence regarding the effectiveness of bone density measurement (BDM) screening and related interventions--hormone replacement therapy (HRT) and intranasal salmon calcitonin (SCT[N])--in menopausal women to prevent fractures in later life. Data sources included systematic reviews of evidence and relevant primary studies identified through literature searches on MEDLINE and EMBASE. Study selection included trials of BDM screening programs, prospective studies examining the predictive value of BDM, randomized controlled trials, cohort studies, and case-control studies of HRT and SCT (N). The evidence was evaluated using a classification system incorporating study design and quality. Outcomes were measured in terms of relative risk of fracture for a 1 SD decrease in bone mineral density below the age-adjusted mean, relative risks or odds ratios for fractures associated with treatments, and proportion of hip fractures potentially prevented by BDM screening linked to treatments. Fair evidence from prospective cohort studies suggests that BDM can predict the risk of fractures, but not with high accuracy. Fair evidence from low-quality randomized controlled trials and observational studies suggests that HRT and SCT(N) are efficacious in preventing fractures. Good evidence supports the efficacy of these treatments in preserving bone mass, but there is fair evidence that the effect wears off after cessation of therapy. There is little evidence on the impact of screening menopausal women with BDM in association with HRT or SCT(N) treatment. Estimates based on combining existing evidence regarding the predictive value of BDM and efficacy of HRT suggests that 1-7% of hip fractures might be prevented.

International collaboration in health technology assessment: a study of technologies used in management of osteoporosis.

A collaborative study was undertaken by members of the International Network of Agencies for Health Technology Assessment (INAHTA). The evidence of the effectiveness of bone density measurement and selected treatments in preventing fractures in later life was reviewed. There was fair evidence that bone density measurement can predict risk of fractures and that hormone replacement therapy and intranasal salmon calcitonin preserve bone mass and decrease the risk of fractures. However, it was estimated that only 1-7% of hip fractures would be prevented if these technologies were used in a screening program for menopausal women. Results of the assessment were endorsed by 13 INAHTA members, disseminated widely and provided input to policy and further work in this area. The project demonstrated the feasibility of international collaborative health technology assessment.